Pediatric patients undergoing stem cell transplant (SCT) are hospitalized for extended periods and are at high risk for sleep disturbances. In order to begin to address the environmental issues that ...
This is a multicenter study that aims to determine whether the EEG and ET experiments studied in the ABC-CT Phase 1 and ABC-CT Confirmation studies can be successfully used with 3-5-year-old children ...
Friedreich Ataxia is a rare condition that causes damage to the nervous system and muscles. People with Friedreich Ataxia have difficulty walking, lose sensation in their arms and legs, and have ...
This is a phase II study looking at patient response to treatment with the combination dinutuximab, temozolomide, irinotecan, and GM-CSF.
Calcium Release Deficiency Syndrome (CRDS) is a newly discovered genetic arrhythmia syndrome that confers a risk of life-threatening arrhythmias secondary to RYR2 loss-of-function. The International ...
The REACH study is for people with CF who do not take cystic fibrosis transmembrane conductance regulator (CFTR) modulators. The goal of the REACH study is to collect research data, including health ...
In this research study, we want to learn more about the impact of two different local anesthetics (bupivacaine and lidocaine) on patient experience following orthognathic (jaw) surgery. Both ...
The primary objective of the study is to evaluate the long-term safety profile of GTX-102 in participants with Angelman Syndrome (AS)
Despite the increasingly common use of cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies in treating cystic fibrosis (CF), it is still largely unknown whether or not other ...
Despite the increasingly common use of cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies in treating cystic fibrosis (CF), it is still largely unknown whether or not other ...